Pharmac seeking proposals on Gaucher disease ERT supply
Gaucher disease is an autosomal recessive disease of lipid storage which results from the deficiency of a lysosomal enzyme. The severity varies widely; some patients present in childhood with virtually all the complications of Gaucher disease, whereas others remain asymptomatic into the eighth decade of life.
The basic goals of treatment are elimination or improvement of symptoms, prevention of irreversible complications and improvement in overall health and quality of life. In children, an additional goal is the optimisation of growth.
Currently Enzyme Replacement Therapy (ERT) with imiglucerase (Cerezyme supplied by Sanofi Genzyme) is funded in New Zealand for eligible patients via application to the Pharmac Gaucher Panel. Imiglucerase has been funded in New Zealand since July 1999 and there are currently 20 patients receiving funded therapy in New Zealand
There is renewed interest in ERT for Gaucher disease with the arrival of new suppliers to the New Zealand market. Pharmac is currently inviting proposals for the supply of a first line ERT for the treatment of Gaucher disease, which closes on the 8th of September. This is an early fact-finding stage only and further processes including public consultation would need to occur before there could be a change to the supply of ERT in New Zealand.
NZORD supports the introduction of new and innovative treatments for rare disorders in New Zealand, whilst acknowledging that a change in therapy can be difficult and stressful for those affected. We are, however, reassured by the following important safeguards in Pharmac’s process:
- Clinical advice will underpin any decisions that are made
- Proposals must allow the existing treatment, Imiglucerase, to be available to those patients who have a clinical reason for being unable to change to a new ERT
- Proposals must include a 6-month transition period to the new ERT